ESPE Abstracts (2023) 97 P1-298

1Service d'Endocrinologie Pédiatrique, Hôpital Universitaire Enfants Malades, Paris, France. 2Centre Hospitalier de Sens, Sens, France. 3Université de Paris, Paris, France. 4Pediatric Endocrinology, Diabetology and Obesity Unit, Lausanne University Hospital, Lausanne, Switzerland

Background: short stature can lead to emotional and social stress in children and adolescents. We previously demonstrated in a cohort of 74 children that a one-year growth hormone treatment (GHT) is associated with a significant improvement of quality of life (QoL) especially on emotional and social scales, both in general (PedsQL 4.0) and height-specific (QoLiSSY) questionnaires. Whether this improvement is sustained until adult height remains to be documented.

Objectives: to evaluate the long-term changes in general and height-specific QoL associated with GHT in short patients who have reached adult height.

Methods: monocentric, prospective, observational, non-interventional, clinical study including the patients from the original cohort who reached adult height (growth velocity <2 cm/year and/or bone age ≥15 years for girls or ≥17 years for boys). Patients and families were recontacted in order to perform the same QoL questionnaires. Change in QoL were evaluated by relative variation and tested by Wilcoxon rank test, each patient being his own control.

Results: Fifty-nine out of 74 children had reached adult height. Among them 10 were lost to follow-up and 19 did not return the questionnaires, leaving 20 patients with complete questionnaires (8 boys and 12 girls). Gain in height was in median +1.5 SD (-0.2 to +3.2). Three patients (too young at inclusion) did not have the initial child QoLISSY questionnaire. The initial parent questionnaire was missing in three other patients. Although PedsQL data were not statistically improved from childhood to adulthood, a significant improvement of the QoLiSSY emotional score was observed, of +0.5 SD (-1.6 to +2.4), P=0.031. Similarly, no improvement was noted on the parental PedsQL data, whereas the parental QoLiSSY questionnaires revealed a significant improvement of children’s social score of +0.9 SD (-0.7 to +1.7), P=0.003, emotional score of +1.5 SD (-0.2 to 2.3), P=0.007, and parental concern about their child’s future, of +0.7 SD (-0.7 to +2.7), P=0.037.

Conclusion: even though the size of the study population does not allow robust conclusions, GHT seems to be associated with a sustained improvement of specific height-related QoL dimensions until adult height, such as the emotional score. Our results also confirm the relevance of using a specific QoL scale for a careful assessment of the potential benefits of GHT in such a population, instead of a more generic one.

Volume 97

61st Annual ESPE (ESPE 2023)

The Hague, Netherlands
21 Sep 2023 - 23 Sep 2023

European Society for Paediatric Endocrinology 

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